Quality of Life and Long-term Outcome of Transition-age Patients with Congenital Hypothyroidism Diagnosed by Newborn Screening and Adequately Treated
Trial Snapshot
- Phase
- Not Applicable
- Status
- Enrolling By Invitation
- Sponsor
- Enrollment
- 150
- Locations
- 1
- Primary Endpoint
- Quality of life of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
Study Overview
Brief Summary
The primary objective of this observational study is the evaluation of the quality of life and long-term outccome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening receiving replacement treatment with levothyroxine.
The secondary objective is to relate the results to the form of hypothyroidism, the initial dose of L-T4, treatment adherence and genetics
Detailed Description
The study protocol will be performed in 3 different phases:
Step1:
A) Enrollment of patients meeting the following criteria:
- diagnosis of congenital hypothyroidism through neonatal screening adequately treated with L-T4
- confirmation of the diagnosis of hypothyroidism upon re-evaluation of the diagnosis
- age 16-21 years at the time of enrollment
B) Information interview on the study and signing of informed consent by the patient and/or parent/guardian
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 16 Years to 21 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Diagnosis of congenital hypothyroidism by neonatal screening
- •Start of L-T4 therapy within the first month of life
- •Confirmation of hypothyroidism condition upon reassessment of diagnosis
Exclusion Criteria
- •other chronic diseases and/or genetic syndromes
- •family history of neuropsychiatric pathology
- •familial dyslipidemia
Arms & Interventions
Adolescents aged 16-21 years with congenital hypothyroidism diagnosed by newborn screening
A) Clinical assessment (Weight, Height, BMI, Waist and hip circumference, blood pressure) B) Assessment of body composition by performing bioimpedance analysis C) Assessment of quality of life by administering the SF36 test D) Assessment of cognitive abilities by administering the WAIS IV test E) Assessment of any psychopathological conditions by means of the SCL-90 psychopathological screening questionnaire F) For patients up to 18 years of age: CBCL questionnaire to be administered to parents to assess the patient's behavior
Intervention: SF36 test (Diagnostic Test)
Adolescents aged 16-21 years with congenital hypothyroidism diagnosed by newborn screening
A) Clinical assessment (Weight, Height, BMI, Waist and hip circumference, blood pressure) B) Assessment of body composition by performing bioimpedance analysis C) Assessment of quality of life by administering the SF36 test D) Assessment of cognitive abilities by administering the WAIS IV test E) Assessment of any psychopathological conditions by means of the SCL-90 psychopathological screening questionnaire F) For patients up to 18 years of age: CBCL questionnaire to be administered to parents to assess the patient's behavior
Intervention: WAIS IV test (Diagnostic Test)
Adolescents aged 16-21 years with congenital hypothyroidism diagnosed by newborn screening
A) Clinical assessment (Weight, Height, BMI, Waist and hip circumference, blood pressure) B) Assessment of body composition by performing bioimpedance analysis C) Assessment of quality of life by administering the SF36 test D) Assessment of cognitive abilities by administering the WAIS IV test E) Assessment of any psychopathological conditions by means of the SCL-90 psychopathological screening questionnaire F) For patients up to 18 years of age: CBCL questionnaire to be administered to parents to assess the patient's behavior
Intervention: SCL-90 psychopathological screening questionnaire (Diagnostic Test)
Adolescents aged 16-21 years with congenital hypothyroidism diagnosed by newborn screening
A) Clinical assessment (Weight, Height, BMI, Waist and hip circumference, blood pressure) B) Assessment of body composition by performing bioimpedance analysis C) Assessment of quality of life by administering the SF36 test D) Assessment of cognitive abilities by administering the WAIS IV test E) Assessment of any psychopathological conditions by means of the SCL-90 psychopathological screening questionnaire F) For patients up to 18 years of age: CBCL questionnaire to be administered to parents to assess the patient's behavior
Intervention: CBCL questionnaire (Diagnostic Test)
Adolescents aged 16-21 years with congenital hypothyroidism diagnosed by newborn screening
A) Clinical assessment (Weight, Height, BMI, Waist and hip circumference, blood pressure) B) Assessment of body composition by performing bioimpedance analysis C) Assessment of quality of life by administering the SF36 test D) Assessment of cognitive abilities by administering the WAIS IV test E) Assessment of any psychopathological conditions by means of the SCL-90 psychopathological screening questionnaire F) For patients up to 18 years of age: CBCL questionnaire to be administered to parents to assess the patient's behavior
Intervention: bioimpedance analysis (Diagnostic Test)
Outcomes
Primary Outcomes
Quality of life of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
Time Frame: evaluation of the long term outcomes at the time of enrolment
Administration of the SF36 questionnaire for the evaluation of quality of life in transition-age children with adequately treated congenital hypothyroidism
Auxological outcome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
Time Frame: evaluation of the long term outcomes at the time of enrolment
weigh SDS, height SDS and BMI SDS assessment in transition-age children with adequately treated congenital hypothyroidism
Intellectual outcome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
Time Frame: evaluation of the long term outcomes at the time of enrolment
Assessment of cognitive abilities through the administration of the WAIS IV
Psychopathological condition of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
Time Frame: evaluation of the long term outcomes at the time of enrolment
Assessment of psychopathological conditions through the SCL-90 questionnaire
Behovioral outcome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening on levothyroxine replacement treatment.
Time Frame: evaluation of the long term outcomes at the time of enrolment
Administration of the CBCL questionnaire to parents of patients up to 18 years of age to describe the patient's behavior
Secondary Outcomes
- Correlation between quality of life and type of congenital hypothyroidism(at time of enrollment)
- Correlation between intellectual outcome and type of congenital hypothyroidism(at time of enrollment)
- Correlation between psychopathological evaluation and type of congenital hypothyroidism(at time of enrollment)
- Correlation between behovioral evaluation and type of congenital hypothyroidism(at time of enrollment)
Investigators
Prof.ssa Mariacarolina Salerno
Full Professor
Federico II University